Currently there are no approved DMT for progressive MS and when trials are done, most times the trials are done in Secondary Progressive MS. This is because there are more people with SPMS than PPMS and so trials are easier to recruit and do.
In the United States, the Rare Diseases Act of 2002 defines rare disease strictly according to prevalence, specifically “any disease or condition that affects less than 200,000 persons in the United States, or about 1 in 1,500 people. This definition is essentially like that of the Orphan Drug Act of 1983, a federal law that was written to encourage research into rare diseases and possible cures. In Japan, the legal definition of a rare disease is one that affects fewer than 50,000 patients in Japan, or about 1 in 2,500 people. The European Commission on Public Health defines rare diseases as “life-threatening or chronically debilitating diseases which are of such low prevalence that special combined efforts are needed to address them. The term low prevalence is later defined as generally meaning fewer than 1 in 2,000 people.
MS is no longer going to get orphan status in the US with 250,000 affected and a multi-billion dollar market. If a case could be made for progressive MS there would be less than 200,000 people affected and there is no available treatment.
Should the MS Community have another go at this?